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GTX 102

Drug Profile

GTX 102

Alternative Names: GTX-102- GeneTx Biotherapeutics; GTX102

Latest Information Update: 22 May 2024

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At a glance

  • Originator Texas A&M AgriLife Research; Texas A&M University
  • Developer GeneTx Biotherapeutics; Ultragenyx Pharmaceutical
  • Class Antisense oligonucleotides; Neuroprotectants
  • Mechanism of Action Gene silencing; UBE3A protein expression stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Angelman syndrome
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Angelman syndrome

Most Recent Events

  • 16 May 2024 Ultragenyx Pharmaceutical plans a phase III trial for Angelman syndrome (In adolescents, In children, In adults, In the elderly) in USA, Australia, Canada, France, Germany, Israel, Spain, United Kingdom (Intrathecal), in August 2024 (NCT06415344) (EudraCT2024-510917-14-00)
  • 02 May 2024 Ultragenyx Pharmaceutical announced its intention to conduct end-of-phase II meeting with the US FDA in mid-2024
  • 02 May 2024 Ultragenyx Pharmaceutical announced its intention to conduct meeting with health authorities in second half of 2024
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